"We are eagerly looking forward to applying this methodology to other tumor types in the laboratory and potentially bringing this approach forward for clinical testing in patients," said Jerry Mendell, MD, director, Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital and a faculty member of The Ohio State University College of Medicine. "While there remains significant work to be done both in identifying such miRNAs and optimizing their delivery, our findings highlight the therapeutic promise of this approach."

The findings of therapeutic miRNA gene replacement in HCC has potential for applicability to other types of cancers, as well. The delivery and restoration of miRNA expression via AAV mediated gene transfer of the miRNA may be beneficial to a large number of cancer subtypes.

"This concept of replacing microRNAs that are expressed in high levels in normal tissues but lost in diseases hasn't been explored before," said Josh Mendell, M.D., Ph.D., an associate professor in the McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine. "Our work raises the possibility of a more general therapeutic approach that is based on restoring microRNAs to diseased tissues."

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